Autologous hematopoietic stem cell transplantation in neuromyelitis optica: A registry study of the EBMT Autoimmune Diseases Working Party

Background: Neuromyelitis optica (NMO) is an inflammatory autoimmune disorder of the central nervoussystem, hallmarked by pathogenic anti-aquaporin 4 antibodies. NMO prognosis is worse comparedwith multiple sclerosis.Objective: The European Group for Blood and Marrow Transplantation (EBMT) Autoimmune DiseasesWorking Party (ADWP) conducted a retrospective survey to analyze disease outcome following autologousstem cell transplantation (ASCT).Methods: This retrospective multicenter study assessed the efficacy and safety of ASCT in 16 patientssuffering from refractory NMO reported to the EBMT registry between 2001 and 2011.Results: Fifteen patients were successfully mobilized with cyclophosphamide (Cy) and G-CSF, one withG-CSF alone. All patients received an unmanipulated autologous peripheral blood stem cell graft, afterconditioning with BEAM plus anti-thymocyte globulin (ATG, n = 9 patients), thiotepa-Cy (n = 3) or Cy(200 mg/kg) plus ATG (n = 4).After a median follow-up of 47 months, three of 16 cases were progression and treatment free, while inthe remaining 13 patients further treatments were administered for disability progression or relapse afterASCT. Altogether, relapse-free survival at three and five years was 31% and 10%, respectively, whileprogression-free survival remained 48% at three and five years.Conclusions: In these NMO patients, highly resistant to conventional treatment, ASCT allows for temporarycontrol of the disease, despite a tendency to progress or relapse in the long term.